Research in Neuromuscular Disorders
Program in Clinical and Translational Neuromuscular Research
The Program in Clinical and Translational Research is focused on bridging the gap between clinical care and the basic sciences in neuromuscular disorders. While this program is based within the Division of Neuromuscular Medicine, we are committed to collaboration across many disciplines, such as immunology, genetics and biochemistry. Our primary research focus has been on immune-mediated neuromuscular diseases, such as myasthenia gravis (MG) and inflammatory neuropathies and myopathies (e.g., Guillain-Barré syndrome, Inclusion Body Myositis). Preliminary observations by our group and others showing the benefit of B cell-targeted therapy for MG have compelled us to explore this further. Plans are underway for a prospective trial of rituximab in MG. Biomarker discovery and working to the end of patient-tailored treatment strategy development is our major overall objective.
We are also planning investigation of the hereditary neuropathies (e.g., Charcot Marie Tooth disease). Our program is a part of the Muscle Study Group (MSG), which is a consortium of scientific investigators among academic and research centers committed to the cooperative planning, implementation, analysis and reporting of controlled clinical trials and of other research for muscle and other neuromuscular diseases.